CRISPR-Based Genome Editing in Gene Therapy: Progress, Applications, and Future Directions
Keywords:
CRISPR, Cas9, gene therapy, genome editing, base editing, prime editing, precision medicineAbstract
The development of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) systems has revolutionized molecular biology and therapeutic research. Originally identified as a bacterial immune system, CRISPR/Cas9 has rapidly evolved into a versatile genome-editing platform with unparalleled simplicity and efficiency. Gene therapy, once hindered by technological and ethical barriers, is now experiencing a renaissance due to CRISPR-enabled precise genetic manipulation. This review discusses the molecular mechanisms of CRISPR/Cas systems, advances in delivery platforms, current applications in monogenic and complex diseases, clinical trial progress, challenges such as off-target
effects and immune responses, ethical considerations, and future innovations including base editing, prime editing, and epigenome editing. A comprehensive perspective is provided on how CRISPR technology is reshaping the therapeutic landscape and its potential role in precision medicine.
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